Is treatment with oral risdiplam effective and well-tolerated for infants with spinal muscular atrophy type 1?

J Perinatol. 2022 May;42(5):689-691. doi: 10.1038/s41372-021-01301-5. Epub 2022 Jan 31.

Authors

Sheema Gaffar¹, Rowena Cayabyab², Rangasamy Ramanathan²

Affiliations

¹ Division of Neonatology, Department of Pediatrics, LAC+USC Medical Center, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. [email protected].
² Division of Neonatology, Department of Pediatrics, LAC+USC Medical Center, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.

PMID: 35095098
DOI: 10.1038/s41372-021-01301-5

No abstract available

MeSH terms

Azo Compounds
Humans
Infant
Pyrimidines
Spinal Muscular Atrophies of Childhood* / drug therapy

Substances

Azo Compounds
Pyrimidines
Risdiplam